Aquaporin 4 and neuromyelitis optica

Lancet Neurol. 2012 Jun;11(6):535-44. doi: 10.1016/S1474-4422(12)70133-3. Epub 2012 May 16.


Neuromyelitis optica is an inflammatory demyelinating disorder of the CNS. The discovery of circulating IgG1 antibodies against the astrocyte water channel protein aquaporin 4 (AQP4) and the evidence that AQP4-IgG is involved in the development of neuromyelitis optica revolutionised our understanding of the disease. However, important unanswered questions remain--for example, we do not know the cause of AQP4-IgG-negative disease, how astrocyte damage causes demyelination, the role of T cells, why peripheral AQP4-expressing organs are undamaged, and how circulating AQP4-IgG enters neuromyelitis optica lesions. New drug candidates have emerged, such as aquaporumab (non-pathogenic antibody blocker of AQP4-IgG binding), sivelestat (neutrophil elastase inhibitor), and eculizumab (complement inhibitor). Despite rapid progress, randomised clinical trials to test new drugs will be challenging because of the small number of individuals with the disorder.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Aquaporin 4* / chemistry
  • Aquaporin 4* / immunology
  • Aquaporin 4* / physiology
  • Humans
  • Immunoglobulin G / biosynthesis
  • Immunoglobulin G / physiology
  • Inflammation Mediators / therapeutic use
  • Neuromyelitis Optica* / drug therapy
  • Neuromyelitis Optica* / immunology
  • Neuromyelitis Optica* / pathology
  • Neuromyelitis Optica* / physiopathology


  • Aquaporin 4
  • Immunoglobulin G
  • Inflammation Mediators